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Long-term Safety and Tolerability of Nizubaglustat in Rare Lysosomal Disorders
NCT07885917Phase 3Not yet recruiting
- Sponsor
- Azafaros
- Start
- Dec 2026
- Primary completion
- Apr 2030
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Overview
Phase 3, open-label, single-arm trial of nizubaglustat in Niemann-Pick disease type C (NPC), GM1 gangliosidosis (GM1), GM2 gangliosidosis and 1 more, sponsored by Azafaros. Primary endpoint: Incidence and severity of treatment-emergent adverse events at 4 years. Planned start Dec 2026; status: not yet recruiting.
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Drugs
nizubaglustat
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Conditions
Niemann-Pick disease type C (NPC)GM1 gangliosidosis (GM1)GM2 gangliosidosisGaucher disease type 3
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